For members of the rare disease community, a clinical trial can be a high stakes process. Patients are often willing to participate in anything that will possibly result in an effective treatment or improve future outcomes for their peers, even if they themselves will not benefit on an individual level. Many patients are so invested in progress that they are willing to make personal sacrifices to achieve a greater good. But sacrifices should never be made unnecessarily, and one of the biggest pitfalls of participating in a clinical trial is the potentiality of receiving a placebo instead of the medication being tested.
Many clinical trials require participants to wash out medications they are already taking; in other words, stop taking them for a set period of time until they are no longer detectable in the patient’s system and then for the duration of the trial.
Herein lies one of the most troubling dilemmas of a modern clinical trial design—is it ethical to make a patient cease one treatment that may be having positive effects only to have them take a placebo in its place?
People cite the declaration of Helsinki which says, “In any medical study, every patient–including those of a control group, if any–should be assured of the best proven diagnostic and therapeutic method.” If this is the ethical guideline for carrying out clinical testing, then it can render many cases of placebo use unethical.
The consequences of placebo use can be even more significant when one considers the possibility of receiving placebo in one trial, not receiving any benefit, yet still being ineligible for another trial down the road due to their participation in the former. Clinical trials managed this way can send desperate patients down a tragic course where possible treatments—that are already rare—are unavailable to them.
Placebos can be useful. We are not suggesting that the practice be eliminated entirely, but when it comes to members of the rare disease community, we believe that they should only be used when absolutely necessary. For many rare diseases, where treatments are few and far in between, patients and caregivers are desperate for some hope. They are often eager to participate in trials, even if it means they have to give up a drug with which they have already had some success. We don’t think that any patient or caregiver should be expected to make such a choice.